New Data from a Long-Term Follow-up Study to the OLYMPUS Trial Show Median Duration of Response of Four Years in Patients Who Achieved a Complete Response with JELMYTO®

Rhea-AI Summary

UroGen Pharma (Nasdaq: URGN) reports new data from a long-term follow-up study of the OLYMPUS trial, showing a median duration of response (DOR) of 47.8 months for patients who achieved complete response with JELMYTO (mitomycin) for pyelocalyceal solution. JELMYTO is FDA approved for treating low-grade, upper tract urothelial cancer (LG-UTUC) in adults. The study, published in the Journal of Urology, included 41 patients from the OLYMPUS trial with a median follow-up of 28.1 months. Notably, 20 of these patients entered the long-term follow-up study. The data suggests robust durability in maintaining cancer control. The study's limitations include its post-hoc nature and selection bias. Further research is ongoing through the JELMYTO uTRACT Registry, with 19 sites activated and 191 patients enrolled as of July 10.

Positive

• Median duration of response of 47.8 months for patients achieving complete response with JELMYTO.
• JELMYTO shows robust durability in treating low-grade UTUC.
• Ongoing research through the JELMYTO uTRACT Registry with 19 sites activated and 191 patients enrolled.

Negative

• Study limitations include post-hoc nature and selection bias of the 20 patients enrolled in the long-term follow-up study.

Insights

Medical Research Analyst

The long-term follow-up data for JELMYTO demonstrates significant clinical value with a median duration of response of 47.8 months in patients who achieved complete response. This is particularly meaningful for LG-UTUC treatment, where organ preservation is crucial. The durability of response suggests reduced need for repeated interventions or radical surgery.

The data strengthens JELMYTO's market position as a kidney-sparing treatment option, aligning with AUA/SUO guidelines. While the study has limitations due to its post-hoc nature and potential selection bias with only 20 patients in long-term follow-up, the ongoing uTRACT Registry with 191 enrolled patients will provide additional real-world evidence to support clinical adoption. This could drive increased physician confidence and potentially expand market penetration.

Financial Analyst

The robust durability data strengthens JELMYTO's competitive position in the LG-UTUC market. With nearly 4 years of median response duration, the treatment offers a compelling value proposition that could drive adoption and reimbursement coverage. The ongoing uTRACT Registry, with substantial patient enrollment, will likely generate additional supportive data to reinforce market acceptance.

For UroGen, this positive long-term data could accelerate JELMYTO's revenue growth by increasing physician confidence and patient acceptance. The publication in a prestigious journal adds credibility and could influence treatment guidelines, potentially expanding the addressable market. However, investors should note the relatively small study size and monitor real-world registry data for broader validation.

The Study Results are Published in the Journal of Urology Online

PRINCETON, N.J.--(BUSINESS WIRE)-- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, today highlights results from a long-term follow-up study with JELMYTO (mitomycin) for pyelocalyceal solution, which is FDA approved for the treatment of low-grade, upper tract urothelial cancer (LG-UTUC) in adult patients. Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41, 20 of whom entered the long-term follow-up study), the median duration of response was 47.8 months (median follow-up 28.1 months [95% CI 13.1, 57.5]). The study results are published online in the Journal of Urology.

“The median DOR of 47.8 months in patients who achieved complete response with JELMYTO provides evidence of robust durability in maintaining control of low-grade UTUC over an extended period,” said Phillip Pierorazio, M.D., Chief, Section of Urology, Penn Presbyterian Medical Center, Professor of Surgery, Hospital of the University of Pennsylvania in Philadelphia, P.A., study author and member of the American Urological Association (AUA)/ Annual Society of Urological Oncology (SUO) UTUC Guideline Committee. “The AUA/SUO recommend that urologists use kidney-sparing techniques as the preferred management strategy for patients with low-grade UTUC when possible. JELMYTO, with this evidence for extended durability of complete response, offers the opportunity for durable recurrence-free intervals.”

Of the 71 patients enrolled in OLYMPUS, 41 achieved a complete response after treatment with JELMYTO and had a median duration of response of 47.8 months (95% CI 13.0, not estimable), with median follow-up of 28.1 months (95% CI 13.1, 57.5).

"Previous research indicates that managing relapse and preserving organ function should be the primary treatment goal for LG-UTUC due to the low risk of disease progression,” said Mark Schoenberg, M.D., Chief Medical Officer of UroGen. “We are encouraged by the study's findings providing compelling evidence for favorable long-term durability of JELMYTO as a primary treatment for LG-UTUC, with increasing evidence showing extended response times in some patients."

The analysis has certain limitations, including its post-hoc nature and the inherent selection bias of the 20 patients enrolled in the long-term follow-up study.

To further explore the potential of JELMYTO in treating patients with UTUC, investigators are currently enrolling participants in the JELMYTO uTRACT Registry to gather longitudinal real-world usage data. As of July 10, 19 sites have been activated with 191 patients enrolled.

About JELMYTO

JELMYTO^® (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for the treatment of adult patients with low-grade-UTUC (LG-UTUC). It is approved for adult patients with LG-UTUC. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as UTUC. In the U.S., there are approximately 6,000 - 7,000 new or recurrent LG-UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). Treatment with endoscopic surgery can lead to a high rate of recurrence and relapse.

About UroGen Pharma Ltd.

UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel^® reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat LG-UTUC and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with low-grade non-muscle invasive bladder cancer are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.urogen.com to learn more or follow us on X (Twitter), @UroGenPharma.

APPROVED USE FOR JELMYTO

JELMYTO^® is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

• are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
• are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
• Tell your healthcare provider if you take water pills (diuretic).

How will I receive JELMYTO?

• Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
• You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
• JELMYTO is given to your kidney through a tube called a catheter.
• During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.

After receiving JELMYTO:

• JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
• To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
• Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.

JELMYTO may cause serious side effects, including:

• Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
• Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
• The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1800FDA1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding plans to enroll participants in the JELMYTO uTRACT Patient Registry to gather longitudinal real-world usage data; the estimated patient population and demographics for UTUC; the potential of UroGen’s proprietary RTGel technology to improve therapeutic profiles of existing drugs; and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: prior results may not be indicative of results that may be observed in the future; potential safety and other complications from JELMYTO use in diverse UTUC patient types; and UroGen’s RTGel technology may not perform as expected and we may not successfully develop and receive regulatory approval of any other product that incorporates UroGen’s RTGel technology. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, filed with the SEC on November 6, 2024 (which is available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241126501899/en/

INVESTOR CONTACT:

Vincent Perrone

Senior Director, Investor Relations

vincent.perrone@urogen.com

609-460-3588 ext. 1093

MEDIA CONTACT:

Cindy Romano

Director, Corporate Communications

cindy.romano@urogen.com

609-460-3583 ext. 1083

Source: UroGen Pharma Ltd.

FAQ

What is the median duration of response for JELMYTO in the OLYMPUS trial?

The median duration of response for JELMYTO in the OLYMPUS trial is 47.8 months.

What is the primary treatment goal for low-grade upper tract urothelial cancer (LG-UTUC)?

The primary treatment goal for LG-UTUC is managing relapse and preserving organ function.

How many patients were enrolled in the JELMYTO uTRACT Registry as of July 10?

As of July 10, 191 patients were enrolled in the JELMYTO uTRACT Registry.

What are the study limitations mentioned in the press release?

The study limitations include its post-hoc nature and the selection bias of the 20 patients enrolled in the long-term follow-up study.